On August 6, 2019 Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, reported its consolidated financial results for the second quarter 2019 and reviewed recent business highlights (Press release, Alnylam, AUG 6, 2019, View Source;p=RssLanding&cat=news&id=2405895 [SID1234538201]).

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"Over the past quarter, we’re pleased with the continued strong progress in the global launch of ONPATTRO. We believe that continued commercial execution with ONPATTRO and expected upcoming launches of other products puts us on a path toward attaining self-sustainability in our business, delivering on the promise of RNAi therapeutics for patients around the world," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "During this period we also achieved several key milestones with our late and earlier stage pipeline, including positive Phase 3 results with givosiran. We expect this track record of commercial and R&D execution to continue well into the future. Specifically, as we turn to the second half of 2019, we look forward to pivotal data readouts from two programs – inclisiran and lumasiran – and additional Phase 3 initiations, namely APOLLO-B with patisiran, HELIOS-B with vutrisiran, and ILLUMINATE-C with lumasiran. Each of these planned milestones will bring us closer to achieving our Alnylam 2020 vision of building a multi-product, global biopharmaceutical company that includes a deep clinical pipeline to fuel continued growth and a robust product engine for sustainable and organic innovation for the future, a profile rarely achieved in the biopharmaceutical industry."

"As we approach the one year anniversary of the ONPATTRO approval, we couldn’t be more proud of our commercial execution. We finished the quarter with over 500 patients on commercial therapy, and we expect continued and steady growth in the years to come driven by new patient finding, global expansion, and additional evidence generation in our ATTR amyloidosis franchise," said Barry Greene, President of Alnylam. "We are committed to decreasing the time to diagnosis and treatment for the benefit of patients with hATTR amyloidosis with polyneuropathy, and we see increased utilization of diagnostic programs such as Alnylam Act. With recent approvals in Japan and Canada, and multiple pricing or reimbursement approvals enabling commercial sales in over ten countries across the CEMEA region, we are expanding our global footprint, bringing ONPATTRO to patients internationally and laying the groundwork for planned future launches of other RNAi therapeutics. Finally, we believe that evidence generation for ONPATTRO will continue to demonstrate the potential benefits and differentiated profile of ONPATTRO."

Second Quarter 2019 and Recent Significant Corporate Highlights

Commercial Performance in Second Quarter 2019

Achieved global ONPATTRO net product revenues for the second quarter of 2019 of $38.2 million.
Attained more than 500 patients worldwide on commercial ONPATTRO treatment as of June 30, 2019.
Received marketing authorization approvals for ONPATTRO in Japan and Canada.
Continued progress with market access efforts across the CEMEA region (Canada, Europe, Middle East, and Africa).
Achieved recent reimbursement approvals and favorable ratings from health technology assessment agencies in England, Scotland, Germany, France, Canada, and Sweden, with significant progress in several additional markets.
R&D Highlights

Advanced patisiran (the non-branded name for ONPATTRO), an intravenously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis, with plans to initiate the APOLLO-B Phase 3 study in ATTR amyloidosis with cardiomyopathy in mid-2019.
Presented positive 12-month data from the Global Open-Label Extension (OLE) study, as well as new analyses from the APOLLO Phase 3 study in patients previously treated with tafamidis and results from an indirect treatment comparison of patisiran versus inotersen in hATTR amyloidosis patients with polyneuropathy.
Advanced vutrisiran (ALN-TTRsc02), a subcutaneously administered investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
Continued enrollment in the HELIOS-A Phase 3 study of vutrisiran in hereditary ATTR amyloidosis patients with polyneuropathy.
The Company announces today that it has obtained regulatory alignment on the design of HELIOS-B – a Phase 3 study of vutrisiran in patients with both hereditary and wild-type ATTR amyloidosis cardiomyopathy – which it expects to start in late 2019.
Advanced givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyria (AHP).
Presented positive results from the ENVISION Phase 3 study.
Completed submissions of a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) and a Marketing Authorisation Application (MAA) with the European Medicines Agency (EMA); both agencies have accepted the applications for filing. The FDA also granted the Company’s request for Priority Review and has set an action date of February 4, 2020, under the Prescription Drug User Fee Act (PDUFA). At this time, the FDA is not planning to hold an advisory committee meeting to discuss this application.
Alnylam announces today that it has inititated an Expanded Access Program for givosiran to support requests by Health Care Providers for pre-approval access for AHP patients.
Advanced lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1).
Completed enrollment in the ILLUMINATE-A Phase 3 study of lumasiran in PH1 patients six years of age or older with mild-to-moderate renal impairment, and remain on track to report results by year-end 2019.
Presented complete positive results from the Phase 1/2 clinical study and positive results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran.
Initiated ILLUMINATE-B, a global Phase 3 pediatric study of lumasiran in PH1 patients under six years of age.
Alnylam’s partner, The Medicines Company, reported new results for inclisiran, an investigational RNAi therapeutic in development for the treatment of hypercholesterolemia.
New data included interim results from the ongoing ORION-3 OLE study in patients with atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalents and results from the ORION-2 and -7 studies in patients with homozygous familial hypercholesterolemia (HoFH) and in patients with renal impairment, respectively.
In addition, The Medicines Company announced that the Independent Data Monitoring Committee for ongoing inclisiran Phase 3 clinical trials (ORION 9, 10, and 11) completed its seventh planned review of safety and efficacy data from the ORION trials and recommended that the trials continue without modification. The safety database for inclisiran now provides more than 3,500 patient-years of exposure to an RNAi therapeutic, representing the industry’s most comprehensive body of safety data for an RNA therapeutic.
Alnylam’s partner, Sanofi, reported new results from the Phase 2 OLE study of fitusiran, an investigational RNAi therapeutic in development for the treatment of hemophilia.
Advanced early- and mid-stage RNAi clinical pipeline.
Initiated a Phase 1 study of ALN-AGT, an investigational RNAi therapeutic targeting angiotensinogen (AGT) for the treatment of hypertension in high unmet need populations, including patients with resistant or refractory hypertension, chronic kidney disease or heart failure.
Announced new platform advances, including preclinical results demonstrating oral delivery of GalNAc-conjugated small interfering RNAs (siRNAs) directed to a liver target. Oral delivery could broaden the clinical and commercial opportunities for RNAi therapeutics, which are currently administered with intravenous or subcutaneous dose administration.
Additional Business Highlights

Formed a broad collaboration with Regeneron Pharmaceuticals, Inc. (Regeneron) to discover, develop, and commercialize RNAi therapeutics focused on ocular and CNS diseases.
Concluded the research and option phase of the Company’s 2014 collaboration with Sanofi focused on advancing RNAi therapeutics for rare genetic diseases.
Entered into a collaboration with 23andMe to support the addition of a new Genetic Health Risk report for Hereditary Amyloidosis (TTR-related). Read more about the report here.
Announced senior leadership changes, including the appointment of Kelley Boucher as the Company’s Senior Vice President, Chief Human Resources Officer; and Jeff Poulton as Executive Vice President, Chief Financial Officer, effective August 13.
Upcoming Events

In the second half of 2019, Alnylam intends to:

Initiate the APOLLO-B Phase 3 study of patisiran in ATTR amyloidosis patients with cardiomyopathy in mid-2019.
Launch ONPATTRO in Japan, England, Switzerland, and multiple other countries.
Initiate the HELIOS-B Phase 3 study of vutrisiran in ATTR amyloidosis patients with cardiomyopathy.
Initiate the ILLUMINATE-C Phase 3 study of lumasiran in PH1 patients with severe renal impairment.
Report topline results from the ILLUMINATE-A Phase 3 study of lumasiran in PH1 patients six years of age or older.
In addition, The Medicines Company intends to report initial topline results from the ORION-9, 10, and 11 Phase 3 studies of inclisiran, and assuming positive results, to file an NDA.

Financial Results for the Quarter Ended June 30, 2019

"Alnylam had strong financial performance in the second quarter. We ended with cash and cash equivalents on our balance sheet of approximately $2.0 billion, bolstered by robust ONPATTRO sales as well as $800 million in additional cash received from our recently announced collaboration with Regeneron," said Manmeet Soni, outgoing Chief Financial Officer of Alnylam. "I have thoroughly enjoyed my time as part of the Alnylam team and am confident that the foundation created over the past few years will serve the Company well."

"I am thrilled to be joining an organization with great near- and long-term growth prospects driven by advancing innovative therapies with the potential to transform patients’ lives," said Jeff Poulton, recently appointed Executive Vice President, Chief Financial Officer of Alnylam, effective August 13. "Having supported the profitable globalization of Shire’s business during my tenure there, I look forward to partnering with the business team at Alnylam to develop a roadmap toward financial self-sustainability."

Cash and Investments
At June 30, 2019, Alnylam had cash, cash equivalents and marketable debt securities, and restricted investments, excluding equity securities, of $1.97 billion, as compared to $1.13 billion at December 31, 2018.

In May 2019, Alnylam received an upfront collaboration payment from Regeneron of $400 million. In addition, Regeneron purchased $400 million of Alnylam equity at a price per share of $90.00 (4.44 million common shares).

GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally accepted in the U.S. (GAAP) for the second quarter of 2019 was $219.5 million, or $2.02 per share on both a basic and diluted basis, as compared to a net loss of $163.6 million, or $1.63 per share on both a basic and diluted basis, for the same period in the previous year.

The non-GAAP net loss for the second quarter of 2019 was $198.3 million, or $1.83 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $161.9 million, or $1.61 per share on both a basic and diluted basis for the same period in the previous year.

Reconciling items between GAAP and non-GAAP net loss include stock-based compensation expense, a gain on the change in fair value of a liability obligation related to the sale of common stock to Regeneron, and a gain on a litigation settlement. See "Use of Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP net loss appearing later in this press release.

ONPATTRO Revenues, Net
Net product revenues from sales of ONPATTRO were $38.2 million in the second quarter of 2019.

Net Revenues from Collaborators
Net revenues from collaborators were $6.5 million in the second quarter of 2019 as compared to $29.9 million in the second quarter of 2018.

GAAP and Non-GAAP Research and Development Expenses
GAAP research and development (R&D) expenses were $163.9 million in the second quarter of 2019 as compared to $137.6 million in the second quarter of 2018.

Non-GAAP R&D expenses were $148.6 million in the second quarter of 2019 as compared to $126.0 million in the second quarter of 2018. Non-GAAP R&D expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP R&D expenses appears later in this press release.

GAAP and Non-GAAP Selling, General and Administrative Expenses
GAAP selling, general and administrative (SG&A) expenses were $112.8 million in the second quarter of 2019 as compared to $84.7 million in the second quarter of 2018.

Non-GAAP SG&A expenses were $97.4 million in the second quarter of 2019 as compared to $74.1 million in the second quarter of 2018. Non-GAAP SG&A expenses exclude stock-based compensation expense. A reconciliation between GAAP and non-GAAP SG&A expenses appears later in this press release.

2019 Updated Financial Guidance
Alnylam is updating its 2019 annual non-GAAP R&D expenses to be in the range of $550 to $575 million (previously $550 to $590 million) and non-GAAP SG&A expenses to be in the range of $390 to $400 million (previously $390 million to $410 million). Both non-GAAP R&D and non-GAAP SG&A expenses exclude stock-based compensation expenses.

The Company expects its current cash, cash equivalents, and marketable debt securities will support company operations for multiple years based upon its current operating plan.

Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.

The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in the press release are stock-based compensation expense, a gain on the change in fair value of a liability obligation, and a gain on litigation settlement. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of a gain on the change in fair value of liability obligation and the gain on litigation settlement because the Company believes these items are one-time events occurring outside the ordinary course of the Company’s business.

The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release.

The Company does not provide in this press release a reconciliation of its estimated 2019 non-GAAP R&D and non-GAAP SG&A expense guidance to the comparable GAAP measures because it is not able to estimate 2019 stock-based compensation expense without unreasonable efforts. The Company’s stock-based compensation expense is subject to significant fluctuations from period to period due to variability in the probability of performance-based vesting events for stock options and restricted stock units and changes in the Company’s stock price which materially impact the recognition, timing of expense and fair value of these awards. In addition, the Company believes such reconciliations for its 2019 financial guidance would imply a degree of precision that would be confusing or misleading to investors.

Conference Call Information
Management will provide an update on the Company and discuss second quarter 2019 results as well as expectations for the future via conference call on Tuesday, August 6, 2019 at 8:00 am ET. To access the call, please dial 800-263-0877 (domestic) or 646-828-8143 (international) five minutes prior to the start time and refer to conference ID 9566932. A replay of the call will be available beginning at 11:00 am ET on the day of the call. To access the replay, please dial 888-203-1112 (domestic) or 719-457-0820 (international) and refer to conference ID 9566932.

About ONPATTRO (patisiran)
ONPATTRO is an RNAi therapeutic that is approved by the U.S. Food and Drug Administration

(FDA) for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is

also approved in the European Union for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy by the Japanese Ministry of Health, Labour and Welfare (MHLW). Based on Nobel Prize-winning science, ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR amyloidosis. It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO blocks the production of TTR in the liver, reducing its accumulation in the body’s tissues in order to halt or slow down the progression of the disease.

Important Safety Information
ONPATTRO is a medicine that treats the polyneuropathy caused by an illness called hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). ONPATTRO is used in adults only.

Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, paracetamol, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Adverse Reactions
The most common adverse reactions that occurred in patients treated with ONPATTRO were respiratory-tract infections (29 percent) and infusion-related reactions (19 percent).

About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNAi therapeutic products using LNP technology.

About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

On August 6, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2019 and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 6, 2019, View Source [SID1234538200]).

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"We have just returned from the 2019 HAEA National Patient Summit with more than 1,000 attendees from the U.S. and around the world. Patients’ excitement about BCX7353 was resounding as they told us an oral option with our safety and efficacy profile could change their lives and the lives of their family members with HAE. Our customers want our product, and we cannot wait to deliver it to them next year," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We are on-track to submit an NDA to the FDA in the fourth quarter, followed by regulatory submissions in Europe and Japan in the first quarter of 2020. We are also preparing for the commercial launch of BCX7353 in the U.S. later in 2020, and we look forward to seeing informative clinical data with our oral Factor-D inhibitor, BCX9930, next quarter," Stonehouse added.

Upcoming Key Milestones

HAE Program – BCX7353

Submit a new drug application (NDA) for oral once-daily BCX7353 for the prevention of HAE attacks with the FDA (Q4 2019)

Submit a marketing authorization application for oral once-daily BCX7353 for the prevention of HAE attacks with the European Medicines Agency (EMA) and Japanese Pharmaceuticals and Medical Devices Agency (PMDA) (Q1 2020)

The company now plans to begin ZENITH-2, a Phase 3 clinical trial of oral BCX7353 (750 mg) for the acute treatment of HAE in 2020, pending the completion of its interactions with regulators on the Phase 3 program and additional CMC formulation work on the acute oral formulation. The company had previously planned to begin ZENITH-2 this summer.
Complement Oral Factor D Inhibitor Program – BCX9930

Report results from ongoing Phase 1 trial of BCX9930 (Q4 2019). The Phase 1 data will inform plans for a proof of concept study in PNH patients in 2020.
ALK-2 Inhibitor Program – BCX9250

Begin a Phase 1 clinical trial of BCX9250, an oral ALK-2 kinase inhibitor for treatment of FOP, in healthy subjects (2H 2019)
Recent Corporate Developments

On July 1, 2019, the company announced it had appointed Megan Sniecinski as chief business officer.
On June 27, 2019, the company announced it had begun enrollment of a Phase 1 trial of BCX9930, an oral Factor D inhibitor discovered and developed by BioCryst, for the treatment of complement-mediated diseases. The trial will evaluate the safety and tolerability and characterize the pharmacokinetic and pharmacodynamic profiles of BX9930 in single and multiple ascending doses of BCX9930 in healthy subjects.
On May 21, 2019, the company announced the successful outcome of APeX-2, a Phase 3 randomized, double-blind, placebo-controlled trial of once-daily, oral BCX7353 for the prevention of hereditary angioedema (HAE) attacks.
On May 10, 2019, the company announced the successful outcome of a randomized, placebo-controlled Phase 1 clinical trial to evaluate intravenous (IV) galidesivir in healthy volunteers.
Second Quarter 2019 Financial Results

For the three months ended June 30, 2019, total revenues were $1.4 million, compared to $12.5 million in the second quarter of 2018. The decrease was primarily due to $7.0 million of deferred revenue and a $5.0 million milestone recognized in the second quarter of 2018, both associated with the EMA’s approval of peramivir (ALPIVABTM).

Research and development (R&D) expenses for the second quarter of 2019 increased to $27.7 million from $21.0 million in the second quarter of 2018, primarily due to increased spending as our HAE programs have progressed and our complement-mediated diseases program entered clinical testing. In addition, the company began recognizing stock option expense for two tranches of performance-based options totaling approximately $2.0 million of expense in the second quarter of 2019. While this expense is allocated to both R&D and G&A, it had a more meaningful impact on R&D expenses for the quarter.

General and administrative (G&A) expenses for the second quarter of 2019 decreased to $8.7 million, compared to $9.5 million in the second quarter of 2018. The decrease was primarily due to a $4.9 million reserve recorded in the second quarter of 2018 for concern regarding the collectability of the EMA approval milestone for peramivir, as well as merger-related costs. These decreases were partially offset by an overall increase in G&A expenses as we prepare for the commercial launch of BCX7353 and an increase in legal costs associated with our ongoing Seqirus UK Limited (Seqirus) dispute.

Interest expense was $3.0 million in the second quarter of 2019, compared to $2.2 million in the second quarter of 2018. The increase was primarily associated with enhancements to the company’s secured credit facility in July 2018 and February 2019.

Net loss for the second quarter of 2019 was $37.6 million, or $0.34 per share, compared to a net loss of $18.5 million, or $0.19 per share, for the second quarter of 2018.

Cash, cash equivalents and investments totaled $97.5 million at June 30, 2019, and reflect a decrease from $128.4 million at December 31, 2018. Operating cash use for the second quarter of 2019 was $26.3 million. Net operating cash use for the first six months of 2019 was $53.4 million as compared to $41.3 million for the first six months of 2018.

Financial Outlook for 2019

BioCryst continues to expect net operating cash use to be in the range of $105 to $130 million, and its operating expenses to be in the range of $120 to $145 million. The company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the company’s stock, as well as by the vesting of the company’s outstanding performance-based stock options. Although not in our operating expense guidance above, approximately $3.5 million of stock option expense will be recognized in the remaining two quarters of 2019 associated with the two tranches of performance-based options mentioned above.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 8954869. A live webcast of the call will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 8954869.

On August 6, 2019 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company" or "we") reported its second-quarter 2019 financial results (Press release, Valeant, AUG 6, 2019, View Source [SID1234538199]).

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"Delivering a second consecutive quarter of reported and organic revenue growth, our second quarter 2019 results demonstrate that Bausch Health is clearly pivoting to growth," said Joseph C. Papa, chairman and CEO, Bausch Health. "Bausch + Lomb/International delivered its eleventh consecutive quarter of organic revenue growth, driven by sustained strength in Global Consumer and Global Vision Care; and Salix reported more than $500 million in total quarterly revenue for the first time."

Mr. Papa continued, "Looking to the second half of 2019, we expect a number of catalysts to drive growth across our core business segments as we continue to reduce debt, increase R&D and further grow our newly launched products. In addition, we have raised our full-year revenue and adjusted EBITDA guidance based on our first half performance and our outlook for the second half of the year."

Company Highlights

Executing on Core Businesses and Advancing Pipeline

The Bausch + Lomb/International segment comprised approximately 56% of the Company’s revenue in the second quarter of 2019
___________________________________
1 Please see the tables at the end of this news release for a reconciliation of this and other non-GAAP measures to the nearest comparable GAAP measure.
2 Organic growth/change, a non-GAAP metric, is defined as a change on a period-over-period basis in revenues on a constant currency basis (if applicable) excluding the impact of recent acquisitions, divestitures and discontinuations.

Reported revenue in the Bausch + Lomb/International segment decreased nominally compared to the second quarter of 2018; revenue in this segment grew organically1,2 by 4% compared to the second quarter of 2018, due to an increase in volume across most of the business units, particularly in Global Consumer and Global Vision Care

Delivered eleventh consecutive quarter of organic revenue growth1,2

LOTEMAX SM (Loteprednol Etabonate Ophthalmic Gel) 0.38% for the treatment of postoperative inflammation and pain following ocular surgery was launched in the U.S. in April 2019

Bausch + Lomb ULTRA Multifocal for Astigmatism contact lenses, the first and only multifocal toric lens available as a standard offering in the eye care professional’s fit set, was launched in the U.S. in June 2019

The Salix segment comprised approximately 24% of the Company’s revenue in the second quarter of 2019

XIFAXAN revenue increased by 21% compared to the second quarter of 2018
◦
Entered into a license agreement with the University of California, Los Angeles to develop and commercialize a novel compound for the treatment of non-alcoholic fatty liver disease and non-alcoholic steatohepatitis

Entered into an exclusive license agreement with Mitsubishi Tanabe Pharma to develop and commercialize a late-stage investigational sphingosine 1-phosphate (S1P) modulator for the treatment of inflammatory bowel disease

The Ortho Dermatologics segment comprised approximately 6% of the Company’s revenue in the second quarter of 2019

Revenues in the Global Solta business increased by 41% compared to the second quarter of 2018, driven by continued strong demand of Thermage FLX in Asia Pacific following the launch in the region

DUOBRII Lotion for the topical treatment of plaque psoriasis in adults was launched in the U.S. in June 2019

Strategic Capital Allocation and Debt Management

Increased research and development by approximately 24%, or $23 million, compared to the second quarter of 2018

Refinanced $1.5 billion of 2023 Senior Unsecured Notes

Reduced debt by approximately $100 million in the second quarter of 2019

Second-Quarter 2019 Revenue Performance
Total reported revenues were $2.152 billion in the second quarter of 2019, as compared to $2.128 billion in the second quarter of 2018, an increase of $24 million. Excluding the unfavorable impact of foreign exchange of $38 million, the impact of a 2019 acquisition of $17 million and the impact of divestitures and discontinuations of $16 million, revenue grew organically1,2 by 3% compared to the second quarter of 2018, driven by organic growth1,2 in the Bausch + Lomb/International and Salix segments.

On August 6, 2019 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, reports its financial results for the quarter ended June 30, 2019 and provides a corporate update (Press release, BioLineRx, AUG 6, 2019, View Source [SID1234538198]).

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Highlights and achievements during the second quarter 2019 and subsequent period:

Continued to advance multiple clinical trials of its lead therapeutic candidates, BL-8040 and AGI-134, and anticipates top-line data from the triple combination arm of the COMBAT/KEYNOTE-202 pancreatic cancer trial by year-end.

Received approval from the FDA for an Investigational New Drug (IND) application for AGI-134, which will enable expansion of the ongoing Phase 1/2a study, currently being carried out in the UK and Israel, to the US by the first half of 2020.

"BL-8040, our lead drug candidate, is rapidly advancing in multiple promising programs. The triple combination arm of our ongoing COMBAT/KEYNOTE-202 Phase 2 study of BL-8040, KEYTRUDA and chemotherapy in metastatic pancreatic cancer is progressing as planned, and top-line results are expected by year end. AML consolidation also remains an important indication in our BL-8040 development plan, and we, with our partners, continue to progress our BLAST Phase 2b study towards a robust interim analysis. In addition, the GENESIS Phase 3 study in stem cell mobilization, our most advanced indication and most direct path to registration, is progressing as planned with top-line results expected in the second half of next year," stated Philip Serlin, Chief Executive Officer of BioLineRx.

"Regarding our second clinical candidate, the universal anti-cancer vaccine AGI-134, we continue to advance our phase 1/2a clinical trial toward initial safety data later this year, and with the acceptance of our IND, we look forward to the next part of the study, which will assess potential efficacy. Taken together, we believe our broad development pipeline provides multiple opportunities for long-term value creation and we are diligently working toward that goal," Mr. Serlin concluded.

Upcoming Milestones

Second half of 2019

Top-line results from COMBAT/KEYNOTE-202 Phase 2 pancreatic cancer trial

Initial safety results from part 1 of Phase 1/2a trial of AGI-134

Potential interim results from Phase 2b AML consolidation study

Initiation of monotherapy basket arm of Part 2 of Phase 1/2a trial of AGI-134

2020

Progression-free survival (PFS) and overall survival (OS) data from COMBAT/KEYNOTE-202 trial in mid-2020

Top-line results from Phase 3 GENESIS registration trial in stem-cell mobilization in second half of 2020

Financial Results for the Second Quarter Ended June 30, 2019

Research and development expenses for the three months ended June 30, 2019 were $5.3 million, an increase of $0.8 million, or 18%, compared to $4.5 million for the three months ended June 30, 2018. The increase resulted primarily from higher expenses associated with the BL-8040 GENESIS and COMBAT clinical trials. Research and development expenses for the six months ended June 30, 2019 were $9.7 million, an increase of $0.1 million, or 2%, compared to $9.6 million for the six months ended June 30, 2018. The small increase resulted primarily from higher expenses associated with the BL-8040 GENESIS and COMBAT clinical trials, offset by a decrease in expenses related to BL-1230, a project which was terminated, as well as a decrease in payroll and related expenses.

Sales and marketing expenses for the three months ended June 30, 2019 were $0.2 million, a decrease of $0.1 million, or 37%, compared to $0.3 million for the three months ended June 30, 2018. The decrease resulted primarily from a decrease in payroll and related expenses. Sales and marketing expenses for the six months ended June 30, 2019 were $0.5 million, a decrease of $0.4 million, or 43%, compared to $0.9 million for the six months ended June 30, 2018. The decrease resulted primarily from a decrease in payroll and related expenses, including a one-time compensation payment in the 2018 period.

General and administrative expenses for the three months ended June 30, 2019 were $0.9 million, similar to the comparable period in 2018. General and administrative expenses for the six months ended June 30, 2019 were $1.9 million, similar to the comparable period in 2018.

The Company’s operating loss for the three months ended June 30, 2019 was $6.5 million, compared to $5.7 million for the three months ended June 30, 2018. The Company’s operating loss for the six months ended June 30, 2019 was $12.1 million, compared to $12.4 million for the comparable period in 2018.

Non-operating income for the three and six months ended June 30, 2019 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, offset by warrant offering expenses. Non-operating income for the six months ended June 30, 2018 primarily relates to fair-value adjustments of warrant liabilities on the Company’s balance sheet, as well as a capital gain from realization of the investment in iPharma.

Net financial expenses amounted to $0.3 million for the three months ended June 30, 2019 compared to net financial income of $0.3 million for the three months ended June 30, 2018. Net financial expenses for the 2019 period primarily relate to interest paid on loans, offset by investment income earned on bank deposits. Net financial income for the 2018 period primarily relates to investment income earned on bank deposits, offset by losses recorded on foreign currency hedging transactions. Net financial expenses amounted to $0.5 million for the six months ended June 30, 2019 compared to net financial income of $0.3 million for the six months ended June 30, 2018. Net financial expenses for the 2019 period primarily relate to interest paid on loans, offset by investment income earned on bank deposits. Net financial income for the 2018 period primarily relates to investment income earned on bank deposits, offset by losses recorded on foreign currency hedging transactions.

The Company’s net loss for the three months ended June 30, 2019 amounted to $5.5 million, compared with a net loss of $4.8 million for the comparable period in 2018. The Company’s net loss for the six months ended June 30, 2019 amounted to $11.6 million, compared with a net loss of $11.0 million for the comparable period in 2018.

The Company held $35.2 million in cash, cash equivalents and short-term bank deposits as of June 30, 2019.

Net cash used in operating activities was $11.1 million for the six months ended June 30, 2019, compared with net cash used in operating activities of $13.0 million for the six months ended June 30, 2018. The $1.9 million decrease in net cash used in operating activities during the six-month period in 2019, compared to the six-month period in 2018, was primarily the result of changes in operating asset and liability items in the two periods., i.e., a decrease in prepaid expenses and other receivables in 2019 versus an increase in 2018, as well as a higher decrease in accounts payable and accruals in 2018.

Net cash used in investing activities was $3.1 million for the six months ended June 30, 2019, compared to net cash provided by investing activities of $10.8 million for the six months ended June 30, 2018. The changes in cash flows from investing activities relate primarily to investments in, and maturities of, short-term bank deposits and the realization of the investment in iPharma in 2018.

Net cash provided by financing activities was $15.7 million for the six months ended June 30, 2019, compared to net cash provided by financing activities of $2.8 million for the six months ended June 30, 2018. The increase in cash flows from financing activities reflects the underwritten public offering completed in February 2019.

Conference Call and Webcast Information

BioLineRx will hold a conference call today, August 6, 2019 at 10:00 a.m. EDT. To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0644 internationally. The call will also be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until August 8, 2019; please dial +1-888-295-2634 from the U.S. or +972-3-925-5904 internationally.

On August 6, 2019 Dova Pharmaceuticals, Inc. (Nasdaq: DOVA), a pharmaceutical company focused on acquiring, developing, and commercializing drug candidates for diseases where there is a high unmet need, reported its operating and financial results for the second quarter ended June 30, 2019 (Press release, Dova Pharmaceuticals, AUG 6, 2019, View Source [SID1234538197]).

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"The last few months have been a transformational period in Dova’s history, with FDA approval and launch of our thrombopoietin receptor agonist (TPO-RA) DOPTELET for ITP, European approval of DOPTELET for severe thrombocytopenia in adult patients with CLD, and an expanded co-promotion partnership with Salix for CLD. These accomplishments have strengthened our position as a growing leader in the treatment of thrombocytopenia," said Dr. David Zaccardelli, President and Chief Executive Officer of Dova. "Beyond ITP, we look forward to results of our further indication expansion activities, with top-line data from our Phase 3 trial of DOPTELET for the treatment of chemotherapy induced thrombocytopenia (CIT) expected in the first half of 2020."

Second Quarter and Subsequent Highlights

DOPTELET ITP Approval – In late June, the FDA approved a supplemental New Drug Application (sNDA) that expanded the use of DOPTELET to include the treatment of thrombocytopenia in adult patients with ITP who have had an insufficient response to a previous treatment.

DOPTELET ITP Launch – In mid-July, DOPTELET was launched commercially for the ITP indication. A sales force of approximately 60 sales professionals with extensive Hematology/

Oncology experience, are now calling on roughly 6,000 HCPs that represent 96% of the ITP patient potential in the United States.

DOPTELET Differentiating Characteristics in ITP –

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DOPTELET is the only oral TPO-RA approved for use in adult patients with ITP that does not have a boxed warning for hepatotoxicity and does not require routine liver enzyme monitoring.

DOPTELET is the only oral TPO approved for use in adult patients with ITP without food-type restrictions.

DOPTELET, as an oral tablet, does not require weekly visits to a health care provider’s office for subcutaneous injections.

DOPTELET increased platelet counts to >50,000 in 66% of ITP patients by Day 8 of treatment in a Phase 3 trial.

DOPTELET ITP Publications – Dova presented four abstracts on the safety and efficacy of avatrombopag from studies in patients with ITP at the 65th International Society on Thrombosis and Haemostasis (ISTH) Annual Scientific Meeting, held July 6-10, 2019, in Melbourne, Australia. These included efficacy data in patients who had previously received another TPO-RA, and the results of a dosing model study.

DOPTELET CLD MAA – In late June, the EC granted marketing authorization for DOPTELET for the treatment of severe thrombocytopenia in adult patients with CLD who are scheduled to undergo an invasive procedure. Further, Dova continues to advance partnering discussions to commercialize DOPTELET in markets outside the US.

Salix Partnership – Dova strategically expanded its co-promotion partnership for CLD in the United States with Salix. Starting on July 1, 2019, in addition to the gastroenterology, colorectal surgery, and proctology segments, Salix has the exclusive right to co-promote DOPTELET for the CLD indication to the hepatology and interventional radiology segments. Dova will continue to pay Salix a commission based on a percentage of net sales in these specialties, which beginning on July 1, 2019, is expected to be in the mid-thirties. In addition, the co-promotion agreement was extended to September 2023.

Phase 3 CIT Clinical Trial – Dova continues to enroll patients in a Phase 3 trial evaluating the safety and efficacy of DOPTELET for the treatment of CIT and plans to report top-line results in the first half of 2020.

DOPTELET CLD Launch Highlights

Net product sales for DOPTELET were $3.5 million for the second quarter.
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Inventory held by specialty pharmacies in Dova’s contracted network decreased by approximately $450,000 from March 31, 2019 to June 30, 2019.
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Prescription referrals increased approximately 7% in the second quarter of 2019 compared to the first quarter of 2019.
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As expected, sales were negatively affected by competitive pricing pressure and payer restrictions in the second quarter of 2019, which Dova is addressing with the new pricing for DOPTELET implemented mid-July 2019.

Early in the second quarter of 2019, a new marketing strategy for the CLD indication was launched, including revised product positioning, messaging, and a new action-oriented brand campaign.

From launch through June 30, 2019, a total of 1,368 health care professionals have prescribed DOPTELET to their patients, with an increasing number of repeat prescribers.

More than 9,200 calls were conducted, reaching more than 4,000 unique health care providers during the second quarter of 2019.

For prescriptions in the second quarter of 2019 that have gone through the adjudication process with payers, 77% of those prescriptions were approved. On average, the time to decision for a referral was 7.4 business days in the second quarter of 2019.

Second Quarter Financial Results

Net product sales for DOPTELET were $3.5 million for the second quarter of 2019. Dova recognizes revenue when products are delivered to its specialty pharmacy partners.

Dova reported a net loss of $17.1 million for the second quarter of 2019, compared to a net loss of $19.7 million for the same period in 2018.

Cost of product sales for the second quarter of 2019 were $0.4 million, which consisted of the cost of inventory, royalty payments and certain distribution and overhead costs.

Research and development expenses were $4.5 million in the second quarter of 2019, compared to $4.5 million for the same period in 2018.

Selling, general and administrative expenses were $15.5 million in the second quarter of 2019, compared to $18.6 million for the same period in 2018. The second quarter of 2018 saw higher expense levels as a result of the full sales staff being onboard for most of the second quarter of 2018 while in 2019 the full sales staff for the launch of DOPTELET for the ITP indication were hired at the end of the second quarter and early third quarter.

As of June 30, 2019, Dova had $76.8 million in cash and equivalents compared to $104.6 million as of December 31, 2018. Additionally, on May 6, 2019, Dova entered into an amended and restated loan agreement with Silicon Valley Bank that extended the interest only period of the existing $20 million loan facility by 12 months and provided additional potential borrowings of $30 million upon achieving certain clinical and revenue milestones. In July 2019, subsequent to the quarter end, Dova drew $10 million on this loan agreement upon approval in ITP.

Company to Host Conference Call

Dova will host a conference call today, August 6, 2019 at 9:00 a.m. ET to discuss second quarter ended June 30, 2019 financial results as well as recent operational highlights. A question-and-answer session will follow Dova’s remarks.

To participate on the live call, please dial 866-550-8145 (domestic) or +1-430-775-1344 (international) and provide the conference ID 7784728 five to 10 minutes before the start of the call.

A live audio webcast of the call will also be available via the "Investor Relations" page of the Dova website, www.dova.com. Please log on through Dova’s website approximately 10 minutes before the scheduled start time. A replay of the webcast will be archived on Dova’s website for 90 days following the call.

Indication and Important Safety Information
INDICATION
DOPTELET (avatrombopag) is indicated for the treatment of thrombocytopenia in adult patients with:

Chronic liver disease who are scheduled to undergo a procedure.

Chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.
IMPORTANT SAFETY INFORMATION FOR DOPTELET
Warnings and Precautions
DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic and thromboembolic complications in patients with chronic liver disease or chronic immune thrombocytopenia. Portal vein thrombosis has been reported in patients with chronic liver disease, and thromboembolic events (arterial and venous) have been reported in patients with chronic immune thrombocytopenia treated with TPO receptor agonists.
In clinical trials, 0.2% (1/430) of patients with chronic liver disease treated with DOPTELET developed a treatment-emergent event of portal vein thrombosis. In clinical trials in patients with chronic immune thrombocytopenia, 7% (9/128) of patients treated with DOPTELET developed a thromboembolic event.
Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions (Factor V Leiden, Prothrombin 20210A, Antithrombin deficiency or Protein C or S deficiency).
DOPTELET should not be administered to patients with chronic liver disease or chronic immune thrombocytopenia in an attempt to normalize platelet counts. Follow the dosing guidelines to achieve target platelet counts. Monitor patients receiving DOPTELET for signs and symptoms of thromboembolic events and institute treatment promptly.
Contraindications: None
Drug Interactions
Dose adjustments are recommended for patients with chronic immune thrombocytopenia taking moderate or strong dual CYP2C9 and CYP3A4 inducers or inhibitors.
Adverse Reactions
The most common adverse reactions (≥3%) in patients with chronic liver disease were: pyrexia, abdominal pain, nausea, headache, fatigue, and peripheral edema.

The most common adverse reactions (≥10%) in patients with chronic immune thrombocytopenia were: headache, fatigue, contusion, epistaxis, upper respiratory tract infection, arthralgia, gingival bleeding, petechiae, and nasopharyngitis.